News

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…

The prices of three approved preventive treatments — Cinryze, Haegarda, and Takhzyro (lanadelumab) — for hereditary angioedema (HAE) must be reduced by more than half for these medications to be cost-effective. Those are the conclusions of a new report published by the Institute for Clinical and Economic Review,…

Regulatory health agencies in Canada and Switzerland are reviewing BioCryst Pharmaceuticals’ applications for the approval of Orladeyo (berotralstat) in their countries for preventing hereditary angioedema (HAE) attacks in patients ages 12 and older. The applications — to Heath Canada and Swissmedic — followed Orladeyo’s recent approvals in the…

KalVista Pharmaceuticals announced plans to soon begin enrolling patients in KOMPLETE, a Phase 2 clinical trial testing its investigational oral therapy KVD824 in preventing swelling attacks in people with hereditary angioedema (HAE) at sites in Canada, Australia, and the U.K. “Over the past month we have made substantial progress in…

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…

Higher blood levels of molecules involved in blood vessel formation and permeability — Tie-2, Ang-2, and VE-Cadherin — along with sE-selectin, which is involved in vascular inflammation, may help to distinguish different types of angioedema, a study suggests. Notably, Tie-2 may be a biomarker of hereditary angioedema (HAE),…

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats are also available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

Takhzyro (lanadelumab) safely and markedly reduced the frequency of swelling attacks in people with hereditary angioedema (HAE) over 2.5 years, according to results of the HELP open-label extension (OLE) study. “This study supports the use of Takhzyro as a long-term preventative treatment option for those 12 years of…

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research into…